[Safety, efficacy, clinical effectiveness, cost-effectiveness and budgetary impact of routine neonatal screening for Pompe disease using tandem mass spectrometry on dried heel-prick blood spots]

Pons-Duran C, Torres-Pagès G, del Toro Riera M, Garcia-Villoria J, Estrada Sabadell MD, Vivanco-Hidalgo RM
Record ID 32018015929
Spanish
Original Title: [Seguridad, eficacia, efectividad clínica, coste-efectividad e impacto presupuestario del cribado neonatal sistemático de la enfermedad de Pompe mediante espectrometría de masas en tándem en sangre seca del talón]
Details
Project Status: Completed
Year Published: 2026
URL for published report: http://hdl.handle.net/11351/14778
English language abstract: An English language summary is available
Publication Type: Full HTA
Country: Spain
MeSH Terms
  • Neonatal Screening
  • Dried Blood Spot Testing
  • Early Diagnosis
  • Mass Screening
  • Infant, Newborn
  • Metabolism, Inborn Errors
  • Citrullinemia
  • Holocarboxylase Synthetase Deficiency
  • Argininosuccinic Aciduria
  • Glycogen Storage Disease Type II
  • Tandem Mass Spectrometry
  • Cost-Effectiveness Analysis
Keywords
  • Pompe Disease
  • tandem mass spectrometry
  • neonatal screening
Contact
Organisation Name: Agència de Qualitat i Avaluació Sanitàries de Catalunya (AQuAS)
Contact Address: Carrer d’Aragó, 330–332 (segona planta) 08009 Barcelona, Spain
Contact Email: atiq.aquas@gencat.cat
This is a bibliographic record of a published health technology assessment from a member of INAHTA or other HTA producer. No evaluation of the quality of this assessment has been made for the HTA database.